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Table 2 Description of simulated disease scenarios assessed fiscally

From: Estimating the fiscal impact of rare diseases using a public economic framework: a case study applied to hereditary transthyretin-mediated (hATTR) amyloidosis

Scenario 1

Early disease onset, at age 45 at PND state 1, with a median progression through PND states, and death at age 55 at PND state 4, without severe cardiomyopathy.

Scenario 2

Early disease onset, at age 45 at PND state 1, with a slow progression through PND states, and death at age 65 at PND state 4, without severe cardiomyopathy.

Scenario 3

Late disease onset, at age 60 at PND state 1, with a median progression through PND states, and death at age 70 at PND state 4, without severe cardiomyopathy.

Scenario 4

Late disease onset and late diagnosis, at age 60 during PND state 2, in a patient with severe cardiomyopathy, where the patient dies within 4 years, at PND state 3b, before reaching PND state 4.